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Shire expands pipeline to treat orphan muscle diseases through collaboration with Acceleron Pharma

Shire plc, the global specialty biopharmaceutical company, today announced the expansion of its Human Genetic Therapies pipeline through the exclusive license, in markets outside of North America, for the activin receptor type IIB (ActRIIB) class of molecules being developed by Acceleron Pharma Inc, a private biotechnology company based in Cambridge, Massachusetts.

The collaboration will initially investigate ACE-031, Acceleron’s lead ActRIIB drug candidate, currently in a Phase 2a trial for the treatment of patients with Duchenne Muscular Dystrophy (DMD). DMD is a fatal orphan muscle disease with no current treatment. ACE-031 and other ActRIIB molecules have the potential to be used in other muscular and neuromuscular disorders with high unmet medical need.

Shire and Acceleron will jointly collaborate on a worldwide development program to advance ACE-031 into a global Phase 2/3 clinical program designed to demonstrate disease modification in DMD patients. Shire will utilise its Lexington, Massachusetts manufacturing facility to produce commercial supplies of the product for both parties. If marketing authorisation is received, Acceleron will commercialise ACE-031 in the U.S. and Canada, and Shire has the exclusive right to commercialise the therapy in the rest of the world.

Under the terms of the agreement, Shire will make an upfront payment to Acceleron of $45 million. Acceleron is eligible to receive additional development, regulatory and sales milestone payments of up to $165 million for the successful commercialisation of ACE-031 in DMD, up to an additional $288 million for successful commercialisation of other indications and molecules, and royalties on product sales. Shire will also make a contribution to global development costs. Shire’s 2010 outlook of Non GAAP earnings trending towards $4.00 per ADS which was recently announced in its second quarter earnings press release is unchanged.

“This collaboration is an excellent strategic fit to the work that Shire is already doing on behalf of patients with rare diseases,” said Sylvie Grégoire, President of Shire Human Genetic Therapies. “Working with Acceleron on the development of ACE-031 for DMD allows us to use our expertise to help patients suffering from this devastating disease, as well as expand our pipeline into a new therapeutic area.”

“The structure of this collaboration allows Acceleron to retain commercial rights in North America with the opportunity to build a highly valuable business while collaborating with an ideal partner for ACE-031,” said John Knopf, PhD, Chief Executive Officer of Acceleron.

“Shire’s international presence and their proven leadership and dedicated focus on orphan diseases forms the basis for successful collaboration to bring innovative therapies to patients with enormous unmet medical need.”

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